Celebrating the world’s first approved gene replacement therapy for an Inherited Retinal Disorder (IRD)
In the lead up to Rare Diseases Day 28 February, we celebrate five years of LUXTURNA, the world’s first approved gene replacement therapy for an Inherited Retinal Disorder (IRD) condition and one of the first gene replacements for any human disease.
The therapy was first delivered to siblings Rylee and Saman, who were born with Leber congenital amaurosis (LCA), a severe form of IRD that progressively narrows and blurs vision until eventual loss of sight.
Their therapy was delivered five years ago by a collaboration of Luminesce Alliance partners including researchers and clinicians from the Genetic Eye Clinic at Sydney Children’s Hospitals Network (SCHN), the Eye Genetics Research Unit and Stem Cell Medicine Group at the Children’s Medical Research Institute (CMRI), the University of Sydney; and the Save Sight Institute at Sydney Eye Hospital.
Since LUXTURNA’s success as a world-first gene replacement therapy, SCHN researchers have made great strides in paving the way for the treatment of patients with all kinds of retinal dystrophies. In a SCHN Kids Advanced Therapeutics Webinar ‘A new pathway to ocular gene therapy‘, hosted by SCHN Director of Research @paulabray, SCHN experts discuss the various aspects of the introduction of LUXTURNA as a life-changing therapy for an inherited blinding eye condition, including the science, eligibility process, treatment delivery, clinical outcomes and institutional impact.
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